Science has a new breakthrough discovery using gene therapy. Inherited blood disorders, for example, can now be treated with Bluebird Bio’s gene therapy called LentiGlobin. The disadvantage, however, relies on the longevity of its results. It is also in question whether the therapy would work for disorders such as beta-thalassemia and sickle cell disease. Bluebird Bio is currently working on this development as they prepare for this year’s official public release of the therapy.

Last December, two participants under this Cambridge-based biotech underwent gene therapy. Unfortunately, they have relapsed and did not go through another trial. Other patients involved in the process have been able to try gene therapy twice since December, and witnessed significant changes without chronic blood transfusions and rise or fall of hemoglobin levels. According to Bluebird Bio, these patients have been cured.

LentiGlobin is a gene therapy that uses stem cells. These cells are obtained from their blood, instead of the bone marrow just as older LentiGlobin procedures used to be done. This type of gene therapy helps patients by replacing their damaged red blood cells with healthy hemoglobin. Patients diagnosed with sickle red cells are now naturally creating ant-sickling hemoglobin from 32 percent to 62 percent. Moreover, Bluebird Bio’s first patient had the most accurate results with a production of anti-sickling hemoglobin at 62 percent. The total hemoglobin level of their first patient is now equally active and healthy as a normal individual.

Bluebird Bio’s other program, beta-thalassemia, had 8 out of 10 patients that no longer needed any blood transfusions within three years. The Bluebird Bio CEO, Nick Leschly, stated that although investors have been getting impatient, the release for gene therapy or LentiGlobin in beta-thalassemia is still set for later this year. “December is later this year,” he further added.

In the US, however, the FDA is asking for more data regarding Bluebird Bio’s LentiGlobin. There may be a possibility of filing LentiGlobin in the US before completing Northstar-2, the 3rd trial of LentiGlobin.

Treating Spinal Injuries with Gene Therapy

Paralyzed rats have also undergone gene therapy. These tests have shown positive results by obtaining paw and bone strength. The rats were able to carry sugar cubes in order to feed themselves. Elizabeth Bradbury, who led the study at King’s College, claimed promising results in a short period of time. She stated, “In some of the tests we looked at – such as gripping the rungs of a ladder – the treatment worked within one to two weeks.” However, lifting, gripping objects, and use of muscles were improved around five to six weeks.

The injuries given to these rats equally represents injuries in humans due to accidents. Researchers placed an injection on their spine that contained the enzyme chondroitinase. This prevented further connection among the nerves, therefore damaging the spinal cord. However, once these tissues have been broken down, the nerves replenish and restore muscle strength.

DNA structure/ Photo By vchal via Shutterstock

Bradbury mentioned in her recent interview that this discovery is extremely helpful as hand movements are the top priority in spinal injuries. Moreover, she mentioned, “Being able to pick up a coffee cup, hold a toothbrush -- these types of things will have a dramatic increase on their quality of life and their independence.”

Can Gene Therapy Save Children from Muscle Dystrophy?

Doctors at the Medical College of Wisconsin are researching on a possible medical breakthrough for muscle disorders. Dr. Michael Lawlor, a neuropathologist and scientist at the Medical College of Wisconsin, claimed this study was also a personal mission for him as he has a close friend diagnosed with muscular dystrophy. There were drastic changes that occurred during their college years. His friend had died from the disease while in medical school.

Since then, Lawlor had focused on this diseases and is now considered as one of the medical doctors to experiment with gene therapy and its beneficial effects toward muscle disorders, especially muscle dystrophy. Dogs have already been tested for the study. Proteins were placed as viruses that increased the dog’s muscle strength. Last year, they were able to test it on patients. Lawlor is now working with two companies with different kinds of diseases to know whether the therapy would work on children.

Lawlor claimed, “The degree of disability many of these children have is extraordinary… if we can get them out of this sort of fragile state, that will be an extraordinary advance." The mother of Lawlor’s friend called the doctor a hero for finding a way to help children with the same disease as her son. For Lawlor, it would not be an easy and quick cure, but rather a breakthrough treatment that can make the lives of these children easier and close to normal.

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